hATTR amyloidosis

FDA approval
#AstraZeneca #FDA #hATTR amyloidosis #Ionis #Phase III trial

Wainua approval marks important milestone in amyloidosis treatment

AstraZeneca and Ionis’ Wainua (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults (hATTR-PN or ATTRv-PN). The approval by the US Food and Drug Administration (FDA) was based on the positive 35-week interim analysis from the… Read More

#News #Regulatory

Polyneuropathy
#European Commission #hATTR amyloidosis #Orphan drugs

RNAi therapeutic approved in EU for hATTR amyloidosis

The European Commission (EC) has granted marketing authorisation for Alnylam Pharmaceuticals’ AMVUTTRA (vutrisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis. The RNAi therapeutic has been approved in adult patients with stage 1 or stage 2 polyneuropathy, following positive 18-month results from the HELIOS-A Phase III study. AMVUTTRA significantly improved… Read More

#News #Regulatory